An Open-Label Pilot Study to Examine the Safety, Tolerability and Efficacy of Deutetrabenazine in Isolated Dystonia.

IF 2.6 4区医学 Q2 CLINICAL NEUROLOGY

Movement Disorders Clinical Practice Pub Date : 2025-01-04 DOI:10.1002/mdc3.14327

Andres Deik, Whitley Aamodt, Christina Cadet, Aaron Lasker, Alexandria Oliver, Meredith Spindler, Thomas F Tropea, Pavan Vaswani, Andrew Siderowf

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引用次数: 0

Abstract

Background: Dystonia may respond to VMAT2 inhibition.

Objectives: Providing pilot data on the safety, tolerability, and efficacy of deutetrabenazine in non dopa-responsive dystonia.

Methods: Deutetrabenazine was titrated by adults with isolated dystonia. Primary study endpoints included the proportion who maintained the individual, maximum tolerated dose for 6 weeks, and how many titrated to 48 mg/day. Secondary endpoints included rates of QTc prolongation/arrhythmias, suicidality, excessive daytime sleepiness, cognitive decline, and drug-induced parkinsonism. Exploratory endpoints for clinical efficacy were assessed.

Results: Among 15 participants, four (26.7%) withdrew early and six (40%) titrated to 48 mg/day. Common adverse events included fatigue and diarrhea. Secondary safety endpoints did not change significantly, but MDS-UPDRS III scores worsened by ≥3 points in seven participants (46.7%). PGI-C and the blinded CGI-C and GDS improved in three women with blepharospasm.

Conclusions: Most participants tolerated deutetrabenazine for 6 weeks, and those with blepharospasm may have benefitted from its use.

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一项开放标签的试点研究，以检查二氘苯那嗪治疗孤立性肌张力障碍的安全性、耐受性和有效性。

背景：肌张力障碍可能对VMAT2抑制有反应。目的：提供deutetrabenazine治疗非多巴反应性肌张力障碍的安全性、耐受性和有效性的先导数据。方法：对成人孤立性肌张力障碍患者进行二苯四嗪滴定。主要研究终点包括维持个体的比例，最大耐受剂量6周，以及滴定至48mg /天的数量。次要终点包括QTc延长/心律失常、自杀倾向、白天过度嗜睡、认知能力下降和药物性帕金森病的发生率。评估临床疗效的探索性终点。结果：在15名参与者中，4名（26.7%）提前退出，6名（40%）滴定至48 mg/天。常见的不良反应包括疲劳和腹泻。次要安全终点无显著变化，但7名参与者（46.7%）的MDS-UPDRS III评分恶化≥3分。3例眼睑痉挛患者的PGI-C、盲法CGI-C和GDS均有改善。结论：大多数参与者耐受deutetrabenazine 6周，眼睑痉挛患者可能受益于该药物的使用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Movement Disorders Clinical Practice CLINICAL NEUROLOGY-

CiteScore

4.00

自引率

7.50%

发文量

218

期刊介绍： Movement Disorders Clinical Practice- is an online-only journal committed to publishing high quality peer reviewed articles related to clinical aspects of movement disorders which broadly include phenomenology (interesting case/case series/rarities), investigative (for e.g- genetics, imaging), translational (phenotype-genotype or other) and treatment aspects (clinical guidelines, diagnostic and treatment algorithms)